Five-year follow-up after gene therapy with ABO-101 in five children with Sanfilippo syndrome type B

Molecular Genetics and Metabolism 2026

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Authors: Mona Lindschau, Yin‐Hsiu Chien, Kevin M. Flanigan, Stefan Nicolau, Kristen V. Truxal, Nicole Muschol

DOI: 10.1016/j.ymgme.2025.109525

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Virus-based gene therapy research Glycogen Storage Diseases and Myoclonus